Golodirsen is an exon-skipping therapy for DMD. Which exon does it target?

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Multiple Choice

Golodirsen is an exon-skipping therapy for DMD. Which exon does it target?

Explanation:
Golodirsen targets exon 53. Exon skipping therapies use an antisense mechanism to remove a specific exon during dystrophin mRNA processing. For certain Duchenne mutations, skipping exon 53 shifts the reading frame back into frame, producing a shorter but functional dystrophin protein that can help stabilize muscle fibers. This exon-specific approach means the drug is intended for patients whose mutations are amenable to skipping exon 53. Other exon-skipping therapies target different exons, such as 45 or 51, depending on the individual mutation, so the exact exon skipped determines which patients may benefit.

Golodirsen targets exon 53. Exon skipping therapies use an antisense mechanism to remove a specific exon during dystrophin mRNA processing. For certain Duchenne mutations, skipping exon 53 shifts the reading frame back into frame, producing a shorter but functional dystrophin protein that can help stabilize muscle fibers. This exon-specific approach means the drug is intended for patients whose mutations are amenable to skipping exon 53. Other exon-skipping therapies target different exons, such as 45 or 51, depending on the individual mutation, so the exact exon skipped determines which patients may benefit.

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